Gene therapy for haemophilia (Q80702723)

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2 January 2020

review article

2 references

29 October 2020

Gene therapy for haemophilia (English)

1 reference

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2 January 2020

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29 October 2020

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29 October 2020

Samuel L Murphy

1

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2 January 2020

Katherine A High

series ordinal

2

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2 January 2020

language of work or name

English

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publication date

1 March 2008

2 references

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2 January 2020

29 October 2020

British Journal of Haematology

2 references

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2 January 2020

29 October 2020

140

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2 January 2020

issue

5

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2 January 2020

page(s)

479-487

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Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer

2 January 2020

cites work

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12 December 2020

Loss of Siglec expression on T lymphocytes during human evolution

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12 December 2020

Bleeding in carriers of hemophilia.

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12 December 2020

Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid

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12 December 2020

Stem cell-derived erythroid cells mediate long-term systemic protein delivery

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12 December 2020

Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.

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12 December 2020

Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes

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12 December 2020

Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets

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12 December 2020

CD8(+) T-cell responses to adeno-associated virus capsid in humans

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12 December 2020

Adenovirus-induced thrombocytopenia: the role of von Willebrand factor and P-selectin in mediating accelerated platelet clearance

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12 December 2020

Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo

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12 December 2020

Epidemiology of adenovirus-associated virus infection in a nursery population

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12 December 2020

A seroepidemiologic study of adenovirus-associated virus infection in infants and children

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12 December 2020

A large region (approximately equal to 95 kDa) of human factor VIII is dispensable for in vitro procoagulant activity

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12 December 2020

Characterization of recombinant human factor VIII

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12 December 2020

In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs

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12 December 2020

Adenovirus mediated expression of therapeutic plasma levels of human factor IX in mice

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12 December 2020

Sustained expression of therapeutic levels of human factor VIII in mice

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12 December 2020

Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector

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12 December 2020

Characterization of the chicken beta-globin insulator

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12 December 2020

Recombinant adeno-associated virus for muscle directed gene therapy

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12 December 2020

Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

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12 December 2020

Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

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12 December 2020

Implantation of autologous skin fibroblast genetically modified to secrete clotting factor IX partially corrects the hemorrhagic tendencies in two hemophilia B patients

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12 December 2020

Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia

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12 December 2020

Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

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12 December 2020

Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

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12 December 2020

Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice

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12 December 2020

Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice

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12 December 2020

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

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12 December 2020

Adenovirus-mediated expression of human coagulation factor IX in the rhesus macaque is associated with dose-limiting toxicity

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12 December 2020

Animal testing of retroviral-mediated gene therapy for factor VIII deficiency

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12 December 2020

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

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12 December 2020

Partial correction of murine hemophilia A with neo-antigenic murine factor VIII.

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12 December 2020

Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy

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12 December 2020

Analysis of testes and semen from rabbits treated by intravenous injection with a retroviral vector encoding the human factor VIII gene: no evidence of germ line transduction

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12 December 2020

Posttranslational modifications of recombinant myotube-synthesized human factor IX.

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12 December 2020

Factors affecting long-term expression of a secreted transgene product after intravenous administration of a retroviral vector

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12 December 2020

Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A.

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12 December 2020

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

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12 December 2020

Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

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12 December 2020

Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy

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12 December 2020

Evaluation of the duration of human factor VIII expression in nonhuman primates after systemic delivery of an adenoviral vector

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12 December 2020

Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques

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12 December 2020

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

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12 December 2020

A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

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12 December 2020

Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors

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12 December 2020

Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice

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12 December 2020

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

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12 December 2020

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

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12 December 2020

Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B.

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12 December 2020

Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion

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12 December 2020

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

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12 December 2020

Intravenous administration of replication-incompetent adenovirus to rhesus monkeys induces thrombocytopenia by increasing in vivo platelet clearance

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12 December 2020

Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A

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12 December 2020

Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.

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12 December 2020

DNA-dependent PK inhibits adeno-associated virus DNA integration

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12 December 2020

Immune responses against adenoviral vectors and their transgene products: a review of strategies for evasion.

1 reference

12 December 2020

Clinical gene transfer studies for hemophilia A.

1 reference

12 December 2020

Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice

1 reference

12 December 2020

Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences

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12 December 2020

Adenoviruses as vaccine vectors

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12 December 2020

Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

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12 December 2020

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

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12 December 2020

Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps

1 reference

12 December 2020

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors

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12 December 2020

Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates.

1 reference

12 December 2020

A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference

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12 December 2020

Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice

1 reference

12 December 2020

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

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12 December 2020

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

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12 December 2020

In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs

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12 December 2020

Stage I clinical trial of gene therapy for hemophilia B.

1 reference

12 December 2020

Successful expression of human factor IX following repeat administration of adenoviral vector in mice.

1 reference

12 December 2020

10.1111/J.1365-2141.2007.06942.X

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:18275425%20AND%20SRC:MED&resulttype=core&format=json

2 January 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:18275425%20AND%20SRC:MED&resulttype=core&format=json

2 January 2020

PubMed publication ID

18275425

1 reference